The company announces that the dosing of patients in its first trial PPCT01, studying the safety and tolerability of XEN-D0501 in patients with type 2 diabetes, was completed.
XEN-D0501 is a highly selective and very potent TRPV1 antagonist, previously in development by Bayer Healthcare and Xention/ Ario Pharma. The TRPV1 target (also called the “chili-receptor”) has demonstrated applications across pain and inflammatory diseases and potentially plays a role in diabetes as well. XEN-D0501 was acquired by PILA PHARMA in 2016, due to its very good safety and tolerability as compared to other clinical TRPV1-antagonist development candidates. TRPV1 antagonists as a drug-class has previously been associated with severe adverse events as fever (hyperthermia). XEN-D0501 has in healthy volunteers been shown to induce a modest temperature increase following the first dose, that fades out during the first 2 weeks of dosing. The maximal tolerable dose in nondiabetic individuals was previously been determined to be 4 mg BID.
In PP-CT01, the doses to be studied were 1, 2 and 4 mg but during the conduct of trial, regulatory approval was obtained to increase to a higher maximal level of 8 mg due to better than expected safety of the lower doses. No serious adverse events were observed in PP-CT01, and only few and mild (expected) adverse events were recorded. Importantly, no hypo-glycemic events nor episodes of hyperthermia were observed.
“I’m very pleased to see, that we did not see any hyperthermia or other severe problems in the diabetic patients,” says Dorte X. Gram, CEO of Pila Pharma. “The finding that no patients experienced hyperthermia and that XEN-D0501 appear to be safe following a single dose to type 2 diabetic patients is really good news”.
A leap forward
The recruitment of patients for PP-CT01 took a lot longer than expected, so in order to not stand still, the company earlier this year applied for the approval to conduct the next pilot phase 2 trial, PP-CT02. In August, the company received the news of regulatory approval of PPCT02 and now works intensively to initiate this pilot phase 2 trial to investigate the efficacy and safety following 28 days dosing of XEN-D0501 BID as compared to placebo in patients with type 2 diabetes.
“It is a tremendous leap forward and could potentially be the steps towards proof-of-concept study in man, that I have been aiming at since the establishment of PILA PHARMA,” says Dorte X. Gram.
Photo of Dorte X. Gram: Jenny Leyman